The US Food and Drug Administration (FDA) has granted Orphan Drug Designation to bexobrutideg (NX-5948), a first-in-class Bruton’s tyrosine kinase (BTK) degrader, for treating Waldenström macroglobulinemia (WM).
Bexobrutideg is an orally bioavailable, brain-penetrant small molecule degrader of BTK that induces ubiquitination and proteasomal degradation, targeting B-cell malignancies with a novel mechanism distinct from inhibitors, according to the therapy’s developer, Nurix. This investigational therapy is being evaluated in a phase 1a/b clinical trial in adults with relapsed or refractory B-cell malignancies.
“The FDA’s Orphan Drug Designation for bexobrutideg, also known as NX-5948, represents an important milestone in our regulatory strategy and underscores the significant unmet medical need for improved treatments for Waldenström macroglobulinemia,” said Arthur T. Sands, MD, PhD, president and CEO of Nurix. “Granting of the designation highlights bexobrutideg’s potential to provide patients with WM a promising new therapeutic option. We are also pleased to announce that our investigational therapy bexobrutideg has been assigned a nonproprietary name reflecting its novel mechanism of action, designated with the unique suffix “deg” for degrader.”
